Key takeaways
- 16 per million DGHO Onkopedia's PNH prevalence estimate for Germany, extrapolated from British and French registries; no domestic count exists
- 1.3 per million new PNH diagnoses annually, same borrowed-estimate basis (DGHO Onkopedia)
- 2 named national referral centres, Ulm and Essen, anchoring German PNH diagnosis and feeding the International PNH Registry
- 19 Dec 2024 G-BA decision date; iptacopan's substantial additional-benefit finding via the orphan pathway
Germany has no domestic PNH prevalence count; DGHO borrows a 16-per-million rate from British and French registries, and the treatment-eligible population routes through just two national referral centres.
Germany is the one market in this model family where the disease-burden layer starts with an explicit gap rather than a number. The DGHO's Onkopedia clinical guideline states plainly that Germany-specific PNH prevalence figures do not exist; the estimate it offers instead, 16 cases per million prevalence and 1.3 new diagnoses per million annually, is extrapolated from British and French registries, not counted domestically. Any epidemiology-based forecast for a German PNH launch inherits the uncertainty of that foreign-registry extrapolation rather than a domestic count. Diagnosis itself requires GPI-anchor flow cytometry, at least two GPI-anchored markers showing deficient or reduced expression across at least two cell lineages, typically granulocytes and reticulocytes, and concentrates at just two named national institutions: the German PNH-Register, housed at Ulm's Institute for Clinical Transfusion Medicine and Immunogenetics, and the West German Cancer Center at University Hospital Essen. Both feed the International PNH Registry, the same dataset underpinning the long-term ravulizumab and eculizumab outcomes literature, but neither publishes an independent German prevalence count of its own.
The treatment-eligible layer is better sourced than the epidemiology layer, because it runs through Germany's AMNOG appraisal process rather than a registry count. Iptacopan, as an orphan-designated therapy, had its additional benefit established through EMA approval alone under paragraph 35a of SGB V, so it skipped a comparator dossier entirely, and the G-BA still found a substantial additional benefit on quality of life on 19 December 2024. Ravulizumab's only PNH-specific G-BA review, a March 2022 assessment of its pediatric indication against eculizumab, found no additional benefit. Crovalimab submitted its benefit dossier on 12 September 2024 and remains under active IQWiG assessment, with no G-BA decision yet. On pricing, no negotiated net price for iptacopan has surfaced in the Lauer-Taxe as of this review, consistent with the manufacturer electing the 2025 MFG confidential-pricing mechanism, a gap with consequences for the 15 countries that reference Germany's published price in their own reimbursement formulas.
Germany PNH funnel — from a borrowed prevalence estimate to the AMNOG-cleared, priced population
| Funnel Stage | Population | Source |
|---|---|---|
| Borrowed PNH prevalence estimate (no domestic count) | 16 per million | DGHO Onkopedia guideline, extrapolated from British and French registries |
| Annual new diagnoses (borrowed estimate) | 1.3 per million | DGHO Onkopedia guideline |
| Named national referral centres | 2 (Ulm, Essen) | German PNH-Register; West German Cancer Center |
| AMNOG-cleared with substantial benefit (orphan pathway) | Iptacopan (Fabhalta) | G-BA Beschluss, 19 Dec 2024 |
Sources: DGHO Onkopedia PNH clinical guideline; German PNH-Register, Institute for Clinical Transfusion Medicine and Immunogenetics, Ulm; West German Cancer Center, University Hospital Essen; G-BA Nutzenbewertungsverfahren Iptacopan (Beschluss 19.12.2024); IQWiG Dossierbewertung Iptacopan (G24-16); G-BA Crovalimab benefit dossier (submitted 12.09.2024); GKV-Spitzenverband negotiation registry.
What this model answers
Every section answers a named commercial question your team is asking, scoped to your asset.
- Why does Germany have no domestic PNH prevalence count, and what does DGHO use instead? • The explicit DGHO Onkopedia statement that Germany-specific figures do not exist • the borrowed 16-per-million prevalence and 1.3-per-million incidence rate extrapolated from British and French registries • what that means for a Germany-specific forecast
- How does the treatment-eligible population route through Germany's AMNOG process, given iptacopan's orphan-pathway benefit finding and crovalimab's pending review? • Iptacopan's 19 Dec 2024 substantial-benefit G-BA decision via the orphan pathway • ravulizumab's unfavourable pediatric-indication HTA record • crovalimab's dossier status under active IQWiG assessment
- What does the live, re-runnable funnel model actually contain, and how is every conversion step sourced? • 8-sheet structure mirroring the standard AXLRx patient flow architecture • DGHO Onkopedia, G-BA, IQWiG, and German PNH-Register citation per conversion step
What's inside
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1 · The Binding Constraint 2 pp
- Why the absence of a domestic prevalence count, not drug access, is the variable a German forecast has to confront first
- Pressure-tested against the DGHO's borrowed British/French registry rate before the rest of the model is built out
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2 · Disease Burden (E1) — Borrowed Prevalence Estimate 3 pp
- 16-per-million prevalence and 1.3-per-million annual incidence, extrapolated from British and French registries (DGHO Onkopedia)
- Why no independently German-counted prevalence figure exists
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3 · Diagnosis & Capture (E2) — Two-Centre Referral Network 4 pp
- German PNH-Register (Ulm) and West German Cancer Center (Essen), the only two named national referral institutions
- GPI-anchor flow cytometry diagnostic criteria and their feed into the International PNH Registry
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4 · Treatment Eligibility (E3) — AMNOG-Cleared Population 3 pp
- Iptacopan's 19 Dec 2024 substantial-benefit G-BA finding via the orphan pathway (SGB V §35a Abs.1 S.11)
- Ravulizumab's unfavourable pediatric-indication HTA record and crovalimab's pending IQWiG dossier
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5 · Market Access (E4) — Pricing & ERP Cascade 3 pp
- The missing Lauer-Taxe net price for Fabhalta and the 2025 MFG confidential-pricing mechanism
- 15-country international reference-price cascade exposure
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6 · Sensitivity Analysis 3 pp
- Which assumption moves the eligible pool most, given no domestic prevalence denominator exists to test against
- Scenario ranges under the public-price versus confidential-price branches
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7 · Year 1·3·5 Projections 4 pp
- Patient volume by horizon under conservative, base, and aggressive uptake scenarios
- Revenue translation inputs under both pricing branches
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8 · Client Alignment Questions 2 pp
- Whether to forecast off DGHO's borrowed rate or commission a Germany-specific epidemiology validation first
- Whether pricing scenarios should assume public Lauer-Taxe disclosure or MFG confidential election
Appendix and source ledger included · 45-minute analyst readout included with delivery
Custom model delivered in 72 hours.
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Commission this model
AXLRx delivers rare disease patient flow models built for forecasting and launch teams sizing the Germany PNH opportunity. Custom model in 72 hours.
- 1. Submit your requestSpecify your indication, market, and cohort definition.
- 2. Scoping callAXLRx analyst confirms funnel scope and comparator set before building.
- 3. DeliveryResearch-verified patient flow model in 72 hours with optional analyst readout.
How AXLRx builds this model
Prepared by MoatRx analysts on the AXLRx commercial consulting engine.
Every AXLRx patient flow model is built on a five-layer funnel: population, disease burden (E1), diagnosis and specialist capture (E2), treatment and biomarker eligibility (E3), market access (E4), then Year 1-3-5 projections across three scenarios. Delivered as a live Excel workbook, not a static table, across 8 sheets with zero hardcoded cells.
Germany PNH sources: the DGHO Onkopedia clinical guideline, the German PNH-Register (Ulm) and West German Cancer Center (Essen), G-BA Nutzenbewertungsverfahren decisions for iptacopan and ravulizumab, IQWiG's Dossierbewertung for iptacopan, and the GKV-Spitzenverband negotiation registry.
- The absence of a domestic German PNH prevalence count, and the borrowed 16-per-million estimate, verified against the DGHO Onkopedia clinical guideline
- The two-centre referral network verified against German PNH-Register (Ulm) and West German Cancer Center (Essen) institutional sources
- Iptacopan's orphan-pathway benefit finding and decision date verified against the live G-BA Beschluss (19 Dec 2024)
- Ravulizumab's pediatric-indication HTA outcome and crovalimab's dossier status verified against the G-BA public record
Frequently asked questions
Deliverables What formats are included with every model?
Every commissioned Patient Flow Model includes an editable 8-sheet Excel funnel model (Strategic Context, Inputs, Model, Projections, Sensitivity, References, Market Context, QC), a PDF methodology brief, and an optional executive readout deck for forecasting and launch team presentations. A 45-minute analyst readout call is included.
Sources How is the epidemiology evidence verified?
AXLRx builds from primary sources only: the DGHO Onkopedia guideline, G-BA and IQWiG appraisal documents, and the German PNH-Register and West German Cancer Center, not secondary market research summaries. Every conversion rate is cited to a primary source and re-runnable in the model.
Customisation Can I tailor the cohort definition or comparator set?
Yes. The intake form captures your indication, target market, cohort definition, and comparators. A scoping call confirms scope before research starts. Commission via the intake form to start.